We spend a lot of “behind the scenes” time working with researchers (both academic and at industry) and an increasing amount of effort affecting policy. We are active in Newborn Screening, FDA policy (PDUFA, FDADSIA, compassionate access, Patient representative Program, etc.), NIH projects (RDCRN, CPAG, NCATS, GRDR, ORDR, etc.), educating and impacting Capital Hill (we can’t “lobby” but our voices are heard loud and clear on funding, sequester, and impacting new legislation), a slew of rare disease activities (registries, biobanks, World Rare Disease Day) and the list goes on.

This blog will be periodically updated to not only help you to know what we are doing but it is designed to educate and inform the general public, those with MLD, and those in the rare disease community. Some postings will be educational, some will solicit a discussion on your thoughts, some will share perspective, and some will be calls to action.

Feel free to leave comments and share your insights and perspective. We’re also interested in hearing what topics you might want to have discussed.